Hematopoietic Stem Cell Transplantation Center Research Articles

Central line-associated bloodstream infection outbreak related to Ralstonia pickettii-contaminated saline in a pediatric hematopoietic stem cell transplant center.

Ralstonia pickettii is an aerobic Gram-negative non-fermentative bacillus. It is an opportunistic pathogen that has recently prompted nosocomial outbreaks. Although it has low virulence, it can cause a wide range of invasive diseases in immunosuppressive patients. The characteristics of R. pickettii-related central line-associated bloodstream infection (CLABSI) outbreak in pediatric hematopoietic stem cell transplant (HSCT) recipients are presented in this study. This was a single-center, retrospective analysis conducted at Bahcesehir University Goztepe Medicalpark Hospital . The clinical and laboratory characteristics of twelve children with Ralstonia-related CLABSIs were analyzed. Of the twelve patients with R. pickettii growth, seven were female. The median age was 12.1 (2-17) years. Autologous HSCT was performed in two of the patients and allogeneic HSCT was performed in ten patients for both malignant and non-malignant diseases. In the conditioning regimens, all patients were given myeloablative therapy. Clinical sepsis was the most common presentation. As a result of the investigations, R. pickettii growth was observed in saline solutions. All cases were successfully treated with the appropriate antibiotic regimen and the bacteria was not found in repeat cultures. Catheter removal was required in two patients. Mortality was not observed in any patient as the outcome of the infection episode. The detection and control of the infectious source are critical in pediatric HSCT patients with severe immunosuppression, as medical equipment-related outbreaks can be life-threatening.

A study of pulmonary infectious and non-infectious complications in a pediatric hematopoietic stem cell transplantation (HSCT) center in Iran

Background: Pulmonary complications are important enough to notice in hematopoietic stem cell transplantation (HSCT). The patients undergoing HSCT might have infectious and non-infectious problems associated with morbidity and mortality. The pulmonary complications of HSCT are well-recognized in adults; however, studies on children are limited, especially in Iran. This study was done to evaluate the infectious and non-infectious pulmonary complications and the corresponding factors in children who underwent HSCT. Materials and Methods: This retrospective cohort study included the patients who underwent HSCT in Mofid Children’s Hospital in Tehran, Iran, during the years 2015 -2021. Overall, 144 medical files were evaluated, out of which 128 had undergone HSCT. The extracted data were about underlying diseases, age at transplant, sex, type of HSCT, donor type, cell source, conditioning regimen, graft versus host disease (GVHD) prophylaxis, infectious and non-infectious pulmonary complications, and associated factors. The data analysis was done by the SPSS software version 26. Chi-square, Fisher exact test and regression were also used for the analysis. Results: Infectious and non-infectious pulmonary complications were reported in 26 people (20.3%) and 11 people (8.6%), respectively. Positive coronavirus disease 2019 (COVID-19) PCR was detected only in two patients. Infectious complications were significantly lower in patients with neuroblastoma compared to other underlying diseases (2.7% vs. 27.5%, P = 0.002). These complications were significantly more frequent among those with other HSCT complications compared to those without such complications (34.6% vs. 16.7%, P=0.042). Non-infectious pulmonary complications were significantly higher in boys (13.5%) than in girls (1.9%) (P = 0.024). Conclusion: Due to the high rate of pulmonary infections in bone marrow transplant patients, clear differential diagnosis and diagnostic work are essential.

First 1-Year Results of a New Hematopoietic Stem Cell Transplant Center

First 1-Year Results of a New Hematopoietic Stem Cell Transplant Center

CT-252 First 1-Year Results of a New Hematopoietic Stem Cell Transplant Center

CT-252 First 1-Year Results of a New Hematopoietic Stem Cell Transplant Center

High infection rates and risk‐adapted prevention strategies in contemporary pediatric allogeneic hematopoietic stem cell transplantation

AbstractWith recent changes in hematopoietic stem cell transplantation (HSCT) practices, such as the increasing use of alternative donors and ex vivo T‐cell depletion, how various risk factors interplay and affect the timeline of infections have not been well elucidated. We retrospectively reviewed the first 100 consecutive HSCT from April 2019 to October 2021 in the only pediatric HSCT center in Hong Kong. We found that the vast majority of the allogeneic transplant recipients (69/74, 93.2%) had infections after HSCT, amongst which bacterial, viral, and fungal infection rates were 35.1%, 90.5%, and 9.5%, respectively. In contrast, only 30.8% (8/26) of autologous transplant recipients had infections (rate of bacterial, viral, and fungal infection were 19.2%, 15.4%, and 3.8%, respectively). Human herpesvirus 6 (HHV‐6) and BK virus (BKV) typically occurred early after HSCT, adenovirus (ADV) and varicella zoster virus (VZV) thereafter, and cytomegalovirus (CMV) and Epstein–Barr virus (EBV) throughout the entire 2.5‐year observation period. Ex vivo T‐cell depletion was a general risk factor for viral infection with HHV‐6 (hazard ratio [HR] = 3.03), BKV (HR = 3.36), CMV (HR = 4.45), and EBV (HR = 7.15); all p < 0.02. Cancer in second‐complete remission compared with first‐complete remission was a risk factor for bacterial infection (OR = 6.0, 95% CI = 1.12–32.2, and p = 0.037). Patients with gut graft‐versus‐host disease were at risk for fungal infections (OR = 12.3, 95% CI = 1.33–114.4, and p = 0.027). The infection‐related mortality rate was 10.0%, which occurred only in allogeneic HSCT patients with hematological malignancies receiving cord blood (n = 4) or haploidentical HSCT (n = 6). Collectively, our findings in pediatric patients after contemporary HSCT support both time‐dependent and risk‐adapted measures against infective complications to improve transplant outcome.

Newborn screening for inborn errors of immunity: The status worldwide

BackgroundNewborn screening (NBS) for the early detection of inborn errors of immunity (IEI) has been implemented in a few countries. The objective of this study was to verify the situation and define obstacles to the implementation of NBS worldwide. MethodsA questionnaire was developed by the Inborn Errors of Immunity Committee of the World Allergy Organization (WAO) with 17 questions regarding NBS for IEI in the physician's workplace, NBS test type, problems hindering NBS implementation, reimbursement for IEI therapy, presence of a national IEI registry, referral centers, molecular diagnosis, hematopoietic stem cell transplantation centers, gene therapy, and immunoglobulin replacement therapy. The survey was sent by email once a week to doctors and others associated with WAO and the main immunology societies worldwide as a Google Form™ to be completed during September and October 2021. ResultsTwo hundred twenty-nine questionnaires were completed, of which 216 (94.3%) were completed by physicians. One hundred seventy-six (76.8%) physicians were both allergists and immunologists. The agreement between allergists/immunologists and non-allergists/non-immunologists for the question “Is there NBS for IEI in the country you work in?” was good (κ = 0,64: 95% CI 0.55–0.69). Ninety-eight (42.8%) participants were from Latin America, 35 (15.3%) from North America, 29 (12.6%) from Europe, 18 (7.9%) from Africa, 44 (19.2%) from Asia, and 5 (2.2%) from Oceania. More than half the participants (n = 124, 54.2%) regularly treated patients with IEI, followed by occasional treatment (n = 77, 33.6%), or never (n = 28, 12.2%). Of the respondents, 14.8% reported that their countries performed NBS for IEI, whereas 42.2% reported their countries did not. T-cell receptor excision circles was the most widely used technique in some countries, with 75 (59.9%) for the diagnosis of NBS for IEI, followed by combined use with kappa deleting-recombination excision circles. Only 13 participants (10.3%) underwent neonatal exon screening in their respective countries. Financial and technical issues were among the major obstacles to the implementation of NBS for IEI. ConclusionsThis pilot study showed that few countries have implemented NBS for IEI, despite the presence of immunology referral centers and the availability of hematopoietic stem cell transplantation and intravenous immunoglobulin replacement therapy. The findings highlight the difficulties, mainly financial and technical, hindering wide application of NBS. Sharing experiences, technologies, and resources at the international level can help overcome these difficulties.

Treatment outcome at the pediatric stem cell transplantation center in Syria: A single-center experience.

Hematopoietic stem cell transplantation (HSCT) is a therapeutic approach known for its high success rates in treating various hematologic malignancies, hemoglobinopathies, immune deficiencies, and other disorders. Notably, pediatric HSCT commenced in Syria in 2021 amidst the prevailing crisis. This study aims to assess the demographic and clinical profiles of pediatric patients who underwent stem cell transplantation and to analyze treatment outcomes at Syria's inaugural pediatric HSCT center. This study is a single-center retrospective analysis of 25 pediatric patients who underwent HSCT underage of 14 years in the National Stem Cell Center (HAYAT) in Damascus within the period 2021-2023. The databases were created based on data that were collected from patient medical records. In autologous patients, transplant-related mortality (TRM) was 0%, with 4 (57%) experiencing disease relapse, resulting in the death of one patient. Additionally, 3 (42.8%) of patients remain alive under second-line management. The overall survival rate was 6 (85.7%), and the disease-free survival rate was 16 (88%). In allogeneic patients, TRM was 5.5% (1/18). One allogeneic patient experienced disease relapse and subsequently died. The overall survival rate and disease-free survival rate were 16 (88%). The objective of this study was to assess the outcomes of pediatric HSCT patients who have undergone transplantation thus far. Given the recent initiation of pediatric stem cell transplantation in Syria, our dataset provides a basis for comparison with international hematopoietic stem cell transplantation centers regarding treatment complications and outcomes, notwithstanding the challenges and crises faced within our country.

Epstein-Barr Virus Monitoring after an Allogeneic Hematopoietic Stem Cell Transplant: Review of the Recent Data and Current Practices in Canada.

Epstein-Barr virus-related post-transplantation lymphoproliferative disorder (EBV-PTLD) is a serious complication following hematopoietic stem cell transplantation (HSCT). A pre-emptive strategy using rituximab, which aims to manage patients early at the time of EBV reactivation to avoid PTLD, has been recommended by the most recent ECIL-6 guidelines in 2016. However, there is still a great heterogeneity of viral-load monitoring protocols, targeted patient populations, and pre-emptive treatment characteristics between centers, making precise EBV monitoring recommendations difficult. We conducted a literature review from the most recent publications between 1 January 2015 and 1 August 2023, to summarize the emerging data on EBV-PTLD prevention strategies in HSCT recipients, including the EBV-DNA threshold and use of rituximab. We also present the results of a survey of current practices carried out in 12 of the main HSCT centers across Canada. We confirm that pre-emptive rituximab remains an efficient strategy for EBV-PTLD prevention. However, there is an urgent need to perform prospective, randomized, multicentric trials with larger numbers of patients reflecting current practices to determine the best clinical conduct with regards to rituximab dosing, timing of treatment, and criteria to initiate treatments. Longer follow-ups will also be necessary to assess patients' long-term outcomes.

Effect of allogeneic hematopoietic stem cell transplantation for chronic granulomatous disease in children: A multicentre, retrospective cohort study in China

Chronic granulomatous disease (CGD) in children is a rare primary immunodeficiency disorder that can lead to life-threatening infections and inflammatory complications. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is increasingly being used to treat severe CGD in children. We conducted a multicenter retrospective analysis of children with CGD who were treated with allo-HSCT at four pediatric hematopoietic stem cell transplant centers in China from September 2005 to December 2019. The study included a total of 171 patients (169 males and 2 females). The median age at the time of transplantation was 6.1 (0–16.4) years. Among them, 154 patients had X-linked recessive inheritance caused by CYBB gene mutations, 12 patients were autosomal recessive, 1 patient had DNAH11 and HYDIN gene mutations, and 4 patients had no gene mutations. The median follow-up period was 36.3 (1.9–79) months. All participating patients were applied to myeloablative conditioning (MAC) regimens. The rates of OS, EFS, and GEFS within three years were 87.5%, 85.3%, and 75.2%, respectively. The total graft failure and the total mortality rate were 5.3% and 11.1%. The cumulative incidence of acute GVHD was 53.8% and the incidence of chronic GVHD was 12.9%, The incidence of chronic GVHD was higher for patients who received unrelated donor cord blood stem cell transplantation (UD-CB) (P = 0.001). Chronic GVHD and coinfections are the risk factors for OS and EFS in patients with CGD after receiving allo-HSCT. UD-CB is a risk factor for EFS and the presence of pneumonia before transplantation is a risk factor for OS. In conclusion, through this study, we have demonstrated that allo-HSCT has excellent efficacy in the treatment of CGD in children, especially, RD-haplo is associated with a lower rate of graft failure incidence and mortality than the treatment modalities of other donor type. Therefore, allo-HSCT is strongly recommended when a well-matched donor is available. If a well-matched donor is not available, the HLA-mismatched donor should be carefully evaluated, and the conditioning regimen modified accordingly.

Clinical analysis of 14 patients aged ≤ 50 years with high-risk multiple myeloma treated with allogeneic hematopoietic stem cell transplantation

Objective: To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in young patients with high-risk multiple myeloma (HRMM) and analyzed the factors affecting patient prognosis. Methods: In this retrospective study, we analyzed the clinical data of 14 patients with HRMM with cytogenetic abnormalities or high-risk biological factors who underwent allo-HSCT at the Hematopoietic Stem Cell Transplantation Center of the Institute of Hematology & Blood Diseases Hospital between November 2016 and November 2022. Results: There were seven males and seven females included in the study, with a median age of 39.5 (31-50) years at the time of allo-HSCT. The median number of treatment lines before transplantation was 2 (1-6) . Before allo-HSCT, 42.9% (6/14) of the patients did not achieve complete remission, while 35.7% (5/14) of the patients achieved measurable residual disease positivity. After transplantation, all patients were evaluated for their treatment response, and the overall response rate was 100% (14/14) . All 14 patients successfully underwent allo-HSCT, with median engraftment times for neutrophils and platelets of 11 (10-14) days and 13 (9-103) days, respectively. Acute grade Ⅱ-Ⅳ graft-versus-host disease (GVHD) occurred in five patients (35.7%) , and two patients (14.3%) developed moderate-to-severe chronic GVHD. The median follow-up time after allo-HSCT was 18.93 (4.10-72.53) months, with an expected 2-year transplant-related mortality rate of 7.1% (95% CI 0%-21.1%) and an expected 2-year overall survival rate of 92.9% (95% CI 80.3%-100.0%) . Moreover, the expected 1-year and 2-year progression-free survival rates were 92.9% (95% CI 80.3%-100.0%) and 66.0% (95% CI 39.4%-100.0%) , respectively, and the 2-year cumulative incidence of relapse was 28.9% (95% CI 0%-56.7%) . Upfront allo-HSCT following complete remission after induced therapy and the presence of chronic GVHD might be favorable prognostic factors. Conclusion: allo-HSCT is an effective treatment for improving the prognosis of young patients with HRMM.

Efficacy of Allogeneic Hematopoietic Stem Cell Transplantation in Young and High-Risk Multiple Myeloma Patients

Objective: With the new therapeutic agents developed over last decades, the median survival of patients with multiple myeloma has approached 10 years. However, 15-20% of patients who are classified as High-risk multiple myeloma (HRMM) patients still have a median survival of less than 3 years, which leaves room for improvements. Simultaneously, transplantation related mortality (TRM) has been decreasing since progress of supportive care during these years. Thus, this study aims to evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in young high-risk multiple myeloma (HRMM) patients and to conduct statistical analysis of various prognostic factors in the new era. Method: This retrospective study analyzed clinical data from 14 young multiple myeloma patients with high-risk cytogenetic abnormalities or high-risk disease biological factors who underwent allo-HSCT at the Hematopoietic Stem Cell Transplantation Center of the Institute of Hematology & Blood Diseases Hospital between November 2016 and November 2022. Results: There were 7 males and 7 females involved in this study, with a median age of 39.5 (31- 50) years at the time of transplantation. The median number of treatment lines before transplantation was 2 (1-6). Before transplantation, 42.9% (6/14) of the patients did not achieve complete remission (CR), with 35.7% (5/14) of the patients performing MRD (+). After transplantation, all patients could be evaluated for treatment response, in which the overall response rate was 100% (14/14). All 14 patients successfully underwent transplantation, with median engraftment times for neutrophils and platelets of 11 (10-14) days and 13 (9-103) days, respectively. Acute graft-versus-host disease (GVHD) of grades II-IV occurred in 5 patients (35.7%), and 2 patients (14.3%) developed moderate to severe chronic GVHD. The median follow-up time after transplantation was 18.93 (4.1-72.53) months, with the 2-year TRM of 7.1% (95%CI 0-19.7%) and the expected 2-year overall survival (OS) rate of 92.9% (95% CI 80.3-100%). Meanwhile, the expected 1-year and 2-year progression-free survival rates were 85.7% (95% CI 69.2-100%) and 45.7% (95% CI 20.9-99.9%), respectively, and the presence of chronic GVHD might influence the outcome positively. Conclusion: Allogeneic hematopoietic stem cell transplantation is an effective treatment to improve the prognosis of young and high-risk multiple myeloma patients.

Why and How Should Ethiopia Establish a Stem Cell Transplant Service? A Review Article.

Ethiopia is attempting to reduce cancer-related morbidity and mortality through a strategic national cancer control plan but according to Globocan 2020, hematologic malignancies particularly leukemia and non-Hodgkin's lymphoma rank among the top five leading causes of new cancer incidence and cause of death among all age groups in both sexes. Hematopoietic stem-cell transplantation (HSCT) is an advanced treatment modality that makes the only effective treatment for cancer and non-cancer-related hematologic diseases unresponsive to conventional therapy. Patients who need stem cell transplants must travel to abroad countries to get the treatment. Meanwhile, the Ethiopian National Specialty and Subspecialty Roadmap sets the goal of establishing HSCT centers in 2020-2029 GC, yet leaders and planners must start taking steps to put the setup in place. Setting up an HSCT facility is challenging for developing countries due to the high costs, limited infrastructure, and need for intensive medical staff training; however, several nations have been able to start successful stem cell transplant programs. This review summarizes the basic steps and requirements of the program in light of guidelines recommendations and lessons learned from other developing countries. It also highlights possible cost-effective opportunities, bottlenecks, and areas that will require work and investment to make the objective reality in Ethiopia. Provides key information to assist administrators and policymakers to set priorities in planning and making informed decisions to establish and maintain the service.

A Phenomenologic Study of the Experiences of Pediatric Cancer Survivors After Hematopoietic Stem Cell Transplantation.

To explore the experiences of pediatric cancer survivors in South Korea after hematopoietic stem cell transplantation. 14 survivors of childhood cancer who had undergone hematopoietic stem cell transplantation at a pediatric hematopoietic stem cell transplantation center. Participants underwent in-depth face-to-face or online interviews from October 2020 to January 2021. Data were analyzed following Giorgi's descriptive phenomenologic research method. The following four themes emerged: regaining physical strength, a wish to express the overwhelming burden, cultivating positive thinking through reflection, and living a self-directed life. Pediatric cancer survivors experienced deep appreciation toward surroundings and everyday life, engaged in broader and greater positive thinking, and strived to live a self-directed life despite physical limitations and negative emotions. Interventions enabling pediatric cancer survivors to express challenges and help them recover physical fitness are needed to improve their post-transplantation quality of life. These results can inform healthcare providers, nurses, and the general community to provide high-quality care to cancer survivors.

Analytical evaluation of the cobas® 6800 system for the detection and quantification of BK Virus (BKV) and Epstein Barr Virus (EBV) at a US solid organ and hematopoietic stem cell transplant center

The cobas® EBV and BKV assays are the first FDA-approved, quantitative assays for monitoring posttransplant reactivation of these viruses. In this study, we assessed performance of the fully-automated cobas® assays, compared with Diasorin Molecular ASR, our laboratory developed test, and demonstrated a strong interassay correlation for BK and EBV monitoring.

Hematopoietic Stem Cell Transplantation Activity for Lymphoma: A Multicentric Study By the Transplantation and Cellular Therapy Mexican Working Group

Introduction: Despite recent advances in the treatment of Hodgkin and non-Hodgkin lymphoma, hematopoietic stem cell transplantation (HSCT) remains the cornerstone of therapy for relapsed and refractory (R/R) disease. Furthermore, in Mexico, the use of targeted therapies like check point inhibitors or monoclonal antibodies is restricted by costs. In addition, information regarding outcomes after HSCT is needed but scarce. Thus, we aimed to register transplantation activity for lymphomas in Mexico. The primary objective was to assess overall survival (OS) and Progression-Free Survival (PFS). Secondary outcomes included to describe and compare the conditioning employed and differences between relapsed and refractory patients. Material and methods: We conducted a retrospective analysis performed by Transplantation and Cellular Therapy Mexican working group (TCTMX), including 8 HSCT transplant centers in Mexico. Patients with HL or NHL who received HSCT from 2016 to 2021 were analyzed. Responses were assessed per the Lugano criteria. Pre-transplant status was defined as relapsed if pateints were in complete response (CR) or partial response (PR) after ≥1 line of treatment, and refractory if they had stable (SD) or progressive disease (PD). Demographic and transplantation data were collected, including the type of transplant, conditioning, maintenance, engraftment, relapse, and death. The OS and PFS were estimated using the Kaplan-Meier method. Results: We included 262 patients from 8 HSCT centers around the country. 148 were men, and 114 were women. The median age was 36 years (range, 16-77). There was an increasing number of transplants by year (108 transplants were performed between 2016 and 2018 and 154 between 2019 and 2021). There were 135 (51.5%) NHL and 127 (48.5%) HL patients, n=227 had relapsed and n=35 refractory disease. Response before HSCT was evaluated with PET-CT in 148 cases (56.4%) and CT scan in 114 patients (43.5%). Autologous transplantation was performed in 200 (76.3%) patients, and 62 (23.7%) received allogeneic HSCT. Among allogeneic HSCT recipients, 19 (30.6%) were MSD, 42 (67.7%) were haploidentical, and 1 (1.61%) matched unrelated donor (MUD). Conditioning regimens included BEAM/BEAM-like in 90 (34.4%), melphalan-based in 87 (33.2%), busulfan-based in 36 (13.7%), and 8 (3.1%) received other regimens. Five-year OS was 79.6% for HL (92% for autologous, and 79.5% for allogeneic), and 69% for NHL (79.5% for autologous, and 43% for allogeneic). Five-year EFS was 37% for HL (72% for autologous, and 30% for allogeneic), and 50.9% for NHL (54.4% for autologous, and 38.3% for allogeneic). The main cause of death was relapse or progressive disease (n= 37, 67.2%). For refractory patients, the median OS was 30 months (95%CI: 3.3-56 months), and the median EFS was 30 months (95%CI: 3.5-6.4 months). Median OS and EFS were not reached for those in CR or PR. The OS and EFS were not significantly different between conditioning regimens. Conclusion: Transplant activity for lymphoma in Mexico is increasing. For relapsed patients, autologous transplantation remains the best option, the lack of carmustine was not an obstacle for favorable outcomes. The prognosis for refractory patients was dismal independently of the type of transplant, and the use of new targeted therapies before transplantation is desirable in this population. Developing outcomes registries in Mexico and other Latin American countries is crucial to better guide treatment decisions in our reality. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Epidemiology, Risk Factors and Outcome of Bacterial, Fungal, and Viral Infections in Pediatric Allogeneic and Autologous Hematopoietic Stem Cell Transplantation Recipients

Objectives Infection remains a major cause of morbidity and mortality after hematopoietic stem cell transplant (HSCT). With recent changes in transplant practices, such as the increasing use of alternative donors and T-cell depletion, how various risk factors interplay and affect the timeline of infections have not been well elucidated in children. While the epidemiology and risk factors for viral infections post-HSCT were well described for cytomegalovirus (CMV) and Epstein-Barr virus (EBV), the corresponding information on human herpes virus 6 (HHV-6), adenovirus (ADV) and BK virus (BKV) infection in children is limited. This study aims to review comprehensively the epidemiology, risk factors and outcome of bacterial, viral and fungal infections after contemporary HSCT to inform future infection prophylaxis strategies. Method We retrospectively reviewed the first 100 consecutive HSCT for malignant and non-malignant diseases from April 2019 to October 2021 in the only pediatric HSCT center in Hong Kong. The incidences were recorded and risk factors were analyzed for bacterial, viral and fungal infections and infection-related mortality. Results The vast majority of the allogeneic transplant recipients (69/74, 93.2%) had infections after HSCT, amongst which bacterial, viral and fungal infection rate was 35.1%, 90.5% and 9.5% respectively. In contrast, only 30.8% (8/26) of autologous transplant recipients had infections, and their respective rate of bacterial, viral and fungal infection was 19.2%, 15.4% and 3.8%. HHV-6 and BKV typically occurred early after HSCT, ADV and varicella zoster virus (VZV) thereafter, and CMV and EBV throughout the entire 2.5-year observation period. The cumulative incidences of HHV-6 and BKV were significantly higher in patients receiving umbilical cord blood (UCB) or haploidentical stem cell transplant (p = 0.0001; p <0.0001, respectively) when compared with matched sibling (MSD) or matched unrelated donor (MUD) transplant (Figure 1 and 2). Ex vivo T-cell depletion was a general risk factor for viral infection with HHV-6 (HR = 3.03), BKV (HR = 3.36), CMV (HR = 4.45) and EBV (HR = 7.15); all p < 0.02. Cancer in second- compared with first-complete remission was a risk factor for bacterial infection in multivariate analyses (OR = 6.0, 95% CI = 1.12-32.2, p = 0.037). Patients with gut graft-versus-host disease were at risk for fungal infections (OR = 12.3, 95% CI = 1.33-114.4, p = 0.027). The infection-related mortality (IRM) rate was 10%, which occurred only in allogeneic HSCT patients with hematological malignancies receiving cord blood (n = 4) or haploidentical HSCT (n = 6). History of bacterial infection after HSCT was associated with an increased risk of IRM (HR = 4.13, 95% CI = 1.05-16.3, p = 0.042). Conclusion Not all HSCT recipients are of equal risk to infections and IRM. Our findings in pediatric patients after contemporary HSCT support both time-dependent and risk-adapted measures against infective complications to improve transplant outcome. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Primary Preventive Care of Hematopoietic Stem Cell Transplantation Survivors: Time to Educate and Empower Recipients and Providers

Increasing use of hematopoietic stem cell transplantation (HCT) and improvements in recipient outcomes have led to a steady increase in the number of allogeneic HCT survivors. In addition to complications specific to the transplantation process, HCT recipients are at increased risk of developing cardiovascular disease (CVD) and subsequent neoplasm (SN). Strict surveillance of risk factors for CVD and cancer in the general population is recommended as an essential component of long-term follow-up (LTFU) care of HCT survivors, but implementation of this has been suboptimal. Various models for improving the provision of survivorship care have been proposed, including a hybrid/combined care approach wherein the HCT provider manages transplantation-specific complications and the primary care physician (PCP) provides general medical care, including surveillance and aggressive management of CVD risk factors and screening for SN. This model also offers a practical approach to LTFU care for HCT survivors who live at a distance from the HCT center, which is a reality for many recipients of HCT at The Ottawa Hospital (TOH). As the success of such a hybrid approach to survivorship care depends on the engagement of HCT recipients with their PCP and compliance with recommended general population surveillance, the aim of the present study was to assess the rates of PCP attendance and adherence to recommended preventive medicine interventions in the years immediately before and after HCT. We hypothesized that rates would be suboptimal and planned to use these results as a baseline for an educational initiative aimed at increasing awareness of HCT recipients and their PCPs about embracing preventive survivorship care. This was a single-center cohort study of allogeneic HCT recipients who underwent transplantation at TOH with linkage to population-based health administrative data. Published clinical practice guidelines were used to define recommended screening for CVD risk factors and cancer. The rates of annual PCP visits and utilization of recommended preventive care interventions in the 5 years before and after HCT were calculated for all eligible patients. Between 2014 and 2020, 409 patients with provincial health care coverage underwent allogeneic HCT at TOH. The median patient age was 51 years (range, 15 to 73 years), with a male predominance (60.9%). Approximately one-quarter of recipients did not attend a PCP visit in the 5 years before and after transplantation, and this proportion increased to one- third in the fifth year post-HCT. Among those recipients who were eligible, only 20% to 25% underwent recommended screening for dyslipidemia and diabetes. Cancer screening rates were also low, at 16% to 18% for cervical cancer, 18% to 22% for colon cancer, and 30% to 31% for breast cancer. These results highlight the need to increase awareness of HCT recipients and their PCPs about the risk of developing CVD and SN post-transplantation and to emphasize the potential to mitigate this risk by adhering to recommendations for surveillance to enable prompt intervention. Patient education should incorporate this information and empower HCT survivors to actively engage in their follow-up care and optimize their long-term outcomes.

Pediatric Hematopoietic Stem Cell Transplantation in Lithuania - 20 Years of Progress through Collaboration.

Hematopoietic stem cell transplantation (HSCT) is a complex procedure that is curative for several fatal pediatric malignancies and non-malignant diseases. Despite its complexity, potential toxicity, and high costs HSCT has become a standard procedure worldwide for several decades. Pediatric HSCT programs encounter several specific challenges. The rarity and heterogeneity of primary diseases, result in an almost 10-fold inferior number of pediatric HSCT as compared to adults. In contrast to the adult programs, where autologous HSCT is more common, allogeneic HSCT (that is more complex) prevails in pediatric setting which is underpinned by a higher number of inborn disorders transplanted in early childhood.In Lithuania, the pediatric HSCT program (EBMT CIC* 508) was launched at Vilnius University Hospital Santaros Klinikos in February 2002. Currently, this is the only specialized pediatric HSCT center in Lithuania and in the Baltic countries. Since 2011 it is a reference center for Latvian children who need autologous or allogeneic transplantation.Here we summarize conference proceedings presented at the scientific event “Pediatric hematopoietic stem cell transplantation in Lithuania – 20 years of progress through collaboration”. The meeting held on September 22-23, 2022, in Vilnius and aimed at commemorating 20 years of the launch of the pediatric transplant program in Lithuania. The event pursued sharing the experience in the field of pediatric HSCT in the Baltic countries. Given a very small population in all three Baltic countries, Lithuania, Latvia, and Estonia face an additional challenge in maintaining sufficient transplant volume and gaining experience. Several distinguished speakers from USA, Denmark, Italy, Germany, Spain, UK and Ukraine shared their expertise in the field and emphasized the crucial role of national and international collaboration to achieve progress in the management of this very rare and complex procedure that offers cure for otherwise fatal pediatric conditions.

P1575: DEVELOPMENT OF A RISK PREDICTION MODEL OF SUBSEQUENT BLOOLDSTREAM INFECTION AFTER CARBAPENEM-RESISTANT ENTEROBACTERIACEASE ISOLATED FROM PERIANAL SWABS IN PATIENTS WITH HEMATOLOGICAL DISEASES

Background: Hematological patients are at higher risk of developing carbapenem-resistant Enterobacteriaceae (CRE) bloodstream infection (BSI) and associated with high mortality. Prediction model for a subsequent CRE BSI in hematological patients with CRE isolated from previous perianal swab samples can provide timely and useful target treatment strategies. Aims: To developing a quantified risk prediction model based on the risk factors of subsequent BSI in CRE carriers will help clinicians better identify specific individuals who would truly benefit from early, empiric CRE-targeted treatment. Methods: The data were extracted from patients with CRE isolated from perianal swab samples at the Hematopoietic Stem Cell Transplantation Center of Blood Diseases Hospital, Chinese Academy of Medical Sciences between July 2017 to October 2020 January. Patients who developed subsequent CRE BSI were compared with those who did not develop BSI. Univariate logistic analysis, multivariate logistic analysis and stepwise regression analysis were carried on a variety of clinical factors. Results: A total of 215 cases were included and 29 (13.4%) patients of them with CRE isolated from perianal swab samples developed CRE BSI subsequently. Multivariate analysis showed that C-reactive protein(CRP)﹥30mg/l(OR 10.613，95%CI 2.965~37.985，P=0.000）, perianal infection(OR 6.450, 95%CI 2.223~18.714, P=0.001), concomitant gastrointestinal symptoms (OR 4.175，95%CI 1.476~11.813，P=0.007），age﹥44 years (OR 3.415，95%CI 1.222~9.541，P=0.007）and neutrophil count﹤0.025×109/L (OR 4.583，95%CI 0.939~22.369，P=0.060）were risk factors for CRE BSI in patients with CRE isolated from perianal swab samples (P<0.01). They were included in the Logistic regression model to predict BSI. According to receiver operating characteristic (ROC) curve analysis, the cut-off value of the model was 0.921 (0.851-0.968，P<0.001). Image:Summary/Conclusion: Hematological patients with CRE isolated from perianal swab samples have a relatively low incidence of subsequent BSI but a relatively high risk of death. The risk prediction model based on CRP, perianal infection, concomitant gastrointestinal symptoms, age and valley absolute neutrophil may be used for predicting subsequent CRE BSI in patients with isolated from perianal swab samples and provide timely and effective treatment reference for this kind of patients.

586 - Data Management Training Program of the First Brazilian Hematopoietic Stem Cell Transplant Center Affiliated to the CIBMTR

586 - Data Management Training Program of the First Brazilian Hematopoietic Stem Cell Transplant Center Affiliated to the CIBMTR